[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100525349":3},{"organization":4,"outcomesModule":7,"designInfo":39,"detailedDescription":45,"studyPopulation":11,"armGroups":46,"interventions":67,"overallOfficials":85,"centralContacts":90,"locations":97,"responsibleParty":328,"collaborators":11,"id":330,"slug":331,"hasResults":332,"nctId":333,"briefTitle":334,"officialTitle":335,"acronym":11,"eligibilityCriteria":336,"healthyVolunteers":332,"sex":337,"minAge":338,"maxAge":11,"enrollmentInfo":339,"targetDuration":11,"studyType":342,"phases":343,"briefSummary":346,"conditions":347,"keywords":11,"overallStatus":100,"whyStopped":11,"lastUpdateSubmitDate":349,"lastUpdatePostDateStruct":350,"startDateStruct":353,"completionDateStruct":355,"leadSponsor":357,"locationsCount":358},{"fullName":5,"class":6},"Biocad","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":15,"otherOutcomes":11},[9,13],{"measure":10,"description":11,"timeFrame":12},"Change in FIX activity from baseline",null,"12 months; final assessment - 5 years",{"measure":14,"description":11,"timeFrame":12},"Proportion of subjects with adverse reactions",[16,19,21,23,25,28,31,34,36],{"measure":17,"description":11,"timeFrame":18},"Changes in FIX activity","5 years",{"measure":20,"description":11,"timeFrame":18},"Annualized use of FIX concentrates",{"measure":22,"description":11,"timeFrame":18},"Annualized bleeding rate",{"measure":24,"description":11,"timeFrame":18},"Annualized rate of bleedings requiring therapy with FIX concentrates",{"measure":26,"description":27,"timeFrame":18},"Changes in Haemo-A-QoL (Hemophilia-Specific Quality of Life) scores from baseline","Haemo-A-QoL is a quality of life (QoL) assessment instrument for patients with haemophilia, including the domains of consequences of bleeding, emotional impact, physical functioning, role functioning, treatment concern, and worry. Using scale overall score ranges from 0 to 100.The high score represent low quality of life.",{"measure":29,"description":30,"timeFrame":18},"Changes in EuroQol-5D-3L (European Quality of Life Questionnaire) scores from baseline","The EQ-5D-5L descriptive system of health-related QoL states consists of 5 dimensions (mobility, self-care, usual activities, pain\u002Fdiscomfort, anxiety\u002Fdepression). The EQ-5D-5L VAS overall score ranges from 0 to 100. A higher score is considered to be more favorable.",{"measure":32,"description":33,"timeFrame":18},"Changes in SF-36 (Short Form-36) scores from baseline","SF-36 is a questionnaire for evaluating health-related QoL (Quality of Life). The 36 questions are meant to reflect 8 domains of health, including physical functioning, physical role, pain, general health, vitality, social function, emotional role, and mental health. SF-3 overall score ranges from 0 to 100. The lower the score the more disability.",{"measure":35,"description":11,"timeFrame":18},"Response duration based on FIX activity",{"measure":37,"description":38,"timeFrame":18},"Evaluation of the condition of joints based on the Hemophilia Joint Health Score (HJHS)","Hemophilia Joint Health Score (HJHS) is the assessment system of joints in patients with haemophilia. Each joint (knee, elbow and ankle) receives a numeric score, which can be compared to itself over time to determine whether a joint is showing degeneration. The maximum score for an individual index joint is 20. Gait is scored 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health.",{"allocation":40,"interventionModel":41,"interventionModelDescription":11,"primaryPurpose":42,"observationalModel":11,"timePerspective":11,"maskingInfo":43},"NON_RANDOMIZED","SEQUENTIAL","TREATMENT",{"masking":44,"maskingDescription":11,"whoMasked":11},"NONE","The study design includes ANB-002 dose escalation in at three cohorts. In Cohort 1, the subject is treated with a single dose of ANB-002 (dose 1) administered as an intravenous infusion. Follow-up for the assessment of dose-limiting toxicity (DLT) will be carried out for 28 days. If no DLT events are observed in the subject of Cohort 1, the following subjects will be included in Cohort 1.\n\nIn Cohort 2, the subjects are treated with a single dose of ANB-002 (dose 2). In Cohort 3, the subject are treated with a single dose of ANB-002 (dose 3).\n\nThe decision to continue enrolling in cohorts will be made at the Independent Data Monitoring Committee (IDMC) meeting. After the IDMC makes a decision regarding the dosing subjects, further enrolment to the cohort will be carried out.\n\nBased on the data from the follow-up period of subjects included in Сohorts 1-3, a potential therapeutic dose for further study will be determined and additional patients will be included to recieve this dose.\n\nIn exploratory Cohort 4 patients with anti-AAV5 antibodies and\u002For hepatitis B in anamnesis will be included. These subjects will recieve the dose 3 of ANB-002.\n\nThe total duration of participation of one subjects in the study will be 5 years.",[47,53,58,63],{"label":48,"type":49,"description":50,"interventionNames":51},"Cohort 1","EXPERIMENTAL","Subjects in Cohort 1 will receive ANB-002 (arvenacogene sanparvovec) at a dose 1. Follow-up for the assessment of dose-limiting toxicity (DLT) will be carried out for 28 days. If no DLT events are observed in the subject of Cohort 1, the following subjects will be included in Cohort 1.",[52],"Genetic: ANB-002, dose 1",{"label":54,"type":49,"description":55,"interventionNames":56},"Cohort 2","Subjects in Cohort 2 will receive ANB-002 (arvenacogene sanparvovec) at a dose 2. The decision to continue enrolling in Cohort 1 or dosing in Cohort 2 will be made at the IDMC meeting. After the IDMC makes a decision regarding the dosing in Cohort 2 subjects, the next subject will be included in Cohort 2. If no DLT events are observed in the subject of Cohort 2, the following subjects will be included in Cohort 2.",[57],"Genetic: ANB-002, dose 2",{"label":59,"type":49,"description":60,"interventionNames":61},"Cohort 3","Subjects in Cohort 3 will receive ANB-002 (arvenacogene sanparvovec) at a dose 3. The decision to continue enrolling in Cohort 2 or dosing in Cohort 3 will be made at the IDMC meeting.",[62],"Genetic: ANB-002, dose 3",{"label":64,"type":49,"description":65,"interventionNames":66},"Cohort 4","Exploratory cohort. Subjects in Cohort 4 (with anti-AAV5 antibodies and\u002For hepatitis B in anamnesis) will receive ANB-002 (arvenacogene sanparvovec) at the dose 3.",[62],[68,75,80],{"type":69,"name":70,"description":71,"armGroupLabels":72,"otherNames":73},"GENETIC","ANB-002, dose 1","Adeno-associated viral vector carrying the FIX gene single infusion at dose 1.",[48],[74],"arvenacogene sanparvovec",{"type":69,"name":76,"description":77,"armGroupLabels":78,"otherNames":79},"ANB-002, dose 2","Adeno-associated viral vector carrying the FIX gene single infusion at dose 2.",[54],[74],{"type":69,"name":81,"description":82,"armGroupLabels":83,"otherNames":84},"ANB-002, dose 3","Adeno-associated viral vector carrying the FIX gene single infusion at dose 3.",[59,64],[74],[86],{"name":87,"affiliation":88,"role":89},"Arina V Zinkina-Orikhan, MD","Director of Clinical Development Department, BIOCAD","STUDY_DIRECTOR",[91],{"name":92,"role":93,"phone":94,"phoneExt":95,"email":96},"Anna Eremeeva, MD PhD","CONTACT","+7 (812) 380 49 34","ext. 6963","eremeevaav@biocad.ru",[98,116,130,146,160,174,188,202,211,222,236,250,264,275,286,300,314],{"facility":99,"status":100,"city":101,"state":11,"zip":102,"country":103,"countryCode":104,"cosmosGeoPoint":105,"geoPoint":110,"contacts":111},"Republican Scientific and Practical Center for Radiation Medicine and Human Ecology","RECRUITING","Homyel","246040","Belarus","BY",{"type":106,"coordinates":107},"Point",[108,109],30.9754,52.4345,{"lat":109,"lon":108},[112],{"name":113,"role":93,"phone":114,"phoneExt":11,"email":115},"Dmitrii Novik","+375232336333","novikdk@mail.ru",{"facility":117,"status":100,"city":118,"state":11,"zip":119,"country":103,"countryCode":104,"cosmosGeoPoint":120,"geoPoint":124,"contacts":125},"Minsk Scientific and Practical Center for Surgery, Transplantology and Hematology","Minsk","220089",{"type":106,"coordinates":121},[122,123],27.56653,53.90019,{"lat":123,"lon":122},[126],{"name":127,"role":93,"phone":128,"phoneExt":11,"email":129},"Anatoly Uss","+375172771091","bmtc@mail.ru",{"facility":131,"status":100,"city":132,"state":11,"zip":133,"country":134,"countryCode":135,"cosmosGeoPoint":136,"geoPoint":140,"contacts":141},"State Autonomous Institution for Healthcare \"Chelyabinsk Regional Clinical Hospital\"","Chelyabinsk","454048","Russia","RU",{"type":106,"coordinates":137},[138,139],61.42877,55.1611,{"lat":139,"lon":138},[142],{"name":143,"role":93,"phone":144,"phoneExt":11,"email":145},"Aleksandr Korobkin","+7 (351) 729 86 60","chelokb@mail.ru",{"facility":147,"status":100,"city":148,"state":11,"zip":149,"country":134,"countryCode":135,"cosmosGeoPoint":150,"geoPoint":154,"contacts":155},"State budgetary healthcare institution Leningrad Regional Clinical Hospital","Gatchina","188300",{"type":106,"coordinates":151},[152,153],30.12833,59.57639,{"lat":153,"lon":152},[156],{"name":157,"role":93,"phone":158,"phoneExt":11,"email":159},"Vladimir Vorobyev","+7 (812) 670 18 88","lokb@47lokb.ru",{"facility":161,"status":100,"city":162,"state":11,"zip":163,"country":134,"countryCode":135,"cosmosGeoPoint":164,"geoPoint":168,"contacts":169},"Kuzbass Clinical Hospital named after S.V. Belyaev","Kemerovo","650066",{"type":106,"coordinates":165},[166,167],86.10435,55.35417,{"lat":167,"lon":166},[170],{"name":171,"role":93,"phone":172,"phoneExt":11,"email":173},"Marina Kosinova","+7 (3842) 39 65 33","05-guz-kokb@kuzdrav.ru",{"facility":175,"status":100,"city":176,"state":11,"zip":177,"country":134,"countryCode":135,"cosmosGeoPoint":178,"geoPoint":182,"contacts":183},"Federal State Budgetary Organization of Science \"Kirov Research Institute of Hematology and Blood Transfusions of Federal Medical Biological agency\"","Kirov","610027",{"type":106,"coordinates":179},[180,181],49.65783,58.59809,{"lat":181,"lon":180},[184],{"name":185,"role":93,"phone":186,"phoneExt":11,"email":187},"Margarita Timofeeva","+7 (8332) 25 59 19","niigpk@fmbamail.ru",{"facility":189,"status":100,"city":190,"state":11,"zip":191,"country":134,"countryCode":135,"cosmosGeoPoint":192,"geoPoint":196,"contacts":197},"Federal State Budgetary Institution \"National Medical Research Centre for Hematology\" of the Ministry of Health of Russian Federation (Department of Clinical Diagnostics for Hematology and Hemostasis Disorders)","Moscow","125167",{"type":106,"coordinates":193},[194,195],37.61781,55.75204,{"lat":195,"lon":194},[198],{"name":199,"role":93,"phone":200,"phoneExt":11,"email":201},"Nadezhda Zozulya","+7 (800) 775 05 82","director@blood.ru",{"facility":203,"status":100,"city":190,"state":11,"zip":191,"country":134,"countryCode":135,"cosmosGeoPoint":204,"geoPoint":206,"contacts":207},"Federal State Budgetary Institution \"National Medical Research Centre for Hematology\" of the Ministry of Health of Russian Federation (Department of Traumatology and Reconstructive and Restorative Orthopedics for Patients with Hemophilia)",{"type":106,"coordinates":205},[194,195],{"lat":195,"lon":194},[208],{"name":209,"role":93,"phone":210,"phoneExt":11,"email":201},"Vladimir Zorenko","+7 (495) 613 24 69",{"facility":212,"status":100,"city":190,"state":11,"zip":213,"country":134,"countryCode":135,"cosmosGeoPoint":214,"geoPoint":216,"contacts":217},"Federal Budgetary Institution \"Moscow City Clinical Hospital named after S. P. Botkin\"","125284",{"type":106,"coordinates":215},[194,195],{"lat":195,"lon":194},[218],{"name":219,"role":93,"phone":220,"phoneExt":11,"email":221},"Vadim Ptushkin","+7 (499) 490 03 03","glavbotkin@zdrav.mos.ru",{"facility":223,"status":100,"city":224,"state":11,"zip":225,"country":134,"countryCode":135,"cosmosGeoPoint":226,"geoPoint":230,"contacts":231},"LLC \"Medis\"","Nizhny Novgorod","603137",{"type":106,"coordinates":227},[228,229],44.00205,56.32867,{"lat":229,"lon":228},[232],{"name":233,"role":93,"phone":234,"phoneExt":11,"email":235},"Svetlana Volkova","+7 (831) 215 20 00","info@medisnn.ru",{"facility":237,"status":100,"city":238,"state":11,"zip":239,"country":134,"countryCode":135,"cosmosGeoPoint":240,"geoPoint":244,"contacts":245},"State Novosibirsk Regional Clinical Hospital","Novosibirsk","630087",{"type":106,"coordinates":241},[242,243],82.93175,55.02259,{"lat":243,"lon":242},[246],{"name":247,"role":93,"phone":248,"phoneExt":11,"email":249},"Tatyana Pospelova","+7 (383) 315 99 99","gnokb@oblmed.nsk.ru",{"facility":251,"status":100,"city":252,"state":11,"zip":253,"country":134,"countryCode":135,"cosmosGeoPoint":254,"geoPoint":258,"contacts":259},"Russian Research Institute of Hematology and Transfusiology Federal State Institution of Federal Medical and Biological Agency","Saint Petersburg","191024",{"type":106,"coordinates":255},[256,257],30.31413,59.93863,{"lat":257,"lon":256},[260],{"name":261,"role":93,"phone":262,"phoneExt":11,"email":263},"Sergey Voloshin","+7 (812) 309 79 81","bloodscience@mail.ru",{"facility":265,"status":100,"city":252,"state":11,"zip":266,"country":134,"countryCode":135,"cosmosGeoPoint":267,"geoPoint":269,"contacts":270},"City Polyclinic №37","191186",{"type":106,"coordinates":268},[256,257],{"lat":257,"lon":256},[271],{"name":272,"role":93,"phone":273,"phoneExt":11,"email":274},"Tatyana Andreeva","+7(812) 312 82 43","p37@zdrav.spb.ru",{"facility":276,"status":100,"city":252,"state":11,"zip":277,"country":134,"countryCode":135,"cosmosGeoPoint":278,"geoPoint":280,"contacts":281},"Almazov National Medical Research Centre","197341",{"type":106,"coordinates":279},[256,257],{"lat":257,"lon":256},[282],{"name":283,"role":93,"phone":284,"phoneExt":11,"email":285},"Vladimir Ivanov","+7 (812) 702 37 16","pmu@almazovcentre.ru",{"facility":287,"status":100,"city":288,"state":11,"zip":289,"country":134,"countryCode":135,"cosmosGeoPoint":290,"geoPoint":294,"contacts":295},"Federal State Budgetary Educational Institution of Higher Education \"Samara State Medical University\" of the Ministry of Healthcare of the Russian Federation","Samara","443099",{"type":106,"coordinates":291},[292,293],50.13553,53.20767,{"lat":293,"lon":292},[296],{"name":297,"role":93,"phone":298,"phoneExt":11,"email":299},"Igor Davydkin","+7 (846) 374 91 00","info@samsmu.ru",{"facility":301,"status":100,"city":302,"state":11,"zip":303,"country":134,"countryCode":135,"cosmosGeoPoint":304,"geoPoint":308,"contacts":309},"State Institution \"Komi Republican Oncological Dispensary\"","Syktyvkar","167904",{"type":106,"coordinates":305},[306,307],50.8163,61.66393,{"lat":307,"lon":306},[310],{"name":311,"role":93,"phone":312,"phoneExt":11,"email":313},"Andrey Proydakov","+7 (800) 100 40 28","mail@gukrod.ru",{"facility":315,"status":100,"city":316,"state":11,"zip":317,"country":134,"countryCode":135,"cosmosGeoPoint":318,"geoPoint":322,"contacts":323},"Federal State Budgetary Educational Institution of Higher Education \"Bashkir State Medical University\" of the Ministry of Healthcare of the Russian Federation","Ufa","450008",{"type":106,"coordinates":319},[320,321],55.96779,54.74306,{"lat":321,"lon":320},[324],{"name":325,"role":93,"phone":326,"phoneExt":11,"email":327},"Bulat Bakhirov","+7 (347) 272 41 73","rectorat@bashgmu.ru",{"type":329,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100525349","phase-1-study-of-the-safety-pharmacodynamics-and-efficacy-of-anb-002-in-patients-with-hemophilia-b-safran-100525349",false,"NCT06120582","Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B (SAFRAN)","An Open-label, Non-comparative, Single Dose-Escalation Study of the Safety, Pharmacodynamics and Efficacy of ANB-002 in Patients With Hemophilia B","Inclusion Criteria:\n\n1. Male with hemophilia B.\n2. Age ≥18 years.\n3. FIX activity at screening ≤2% without FIX inhibitor.\n4. ≥150 previous exposure days of treatment with FIX concentrates.\n\nExclusion Criteria:\n\n1. Previous gene therapy.\n2. Other blood or hematopoietic disorders.\n3. Positive Anti-AAV5 antibodies (for Cohorts 1-3).\n4. Diagnosed HIV-infection, not controlled with anti-viral therapy.\n5. Hepatitis B (for Cohorts 1-3), acute or chronic hepatitis C.\n6. Any active systemic infections or recurrent infections requiring systemic therapy at screening.\n7. Any other disorders associated with severe immunodeficiency.\n8. Significant hepatic disorders (liver cirrhosis, liver fibrosis, etc).\n9. Malignancies with remission \\\u003C5 years.","MALE","18 Years",{"count":340,"type":341},28,"ESTIMATED","INTERVENTIONAL",[344,345],"PHASE1","PHASE2","The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-002 in subjects with hemophilia В. The study will have a dose-escalation design with elements of phase I\u002FII seamless adaptive design.",[348],"Hemophilia B","2025-06-10",{"date":351,"type":352},"2025-06-13","ACTUAL",{"date":354,"type":352},"2023-05-02",{"date":356,"type":341},"2029-11",{"name":5,"class":6},17]